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Trial details imported from ClinicalTrials.gov
For full trial details, please see the original record at
https://clinicaltrials.gov/ct2/show/NCT02301624
Registration number
NCT02301624
Ethics application status
Date submitted
22/11/2014
Date registered
26/11/2014
Date last updated
5/02/2020
Titles & IDs
Public title
Extension Study of ECU-MG-301 to Evaluate Safety and Efficacy of Eculizumab in Refractory Generalized Myasthenia Gravis
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Scientific title
A Phase III, Open-label Extension Trial of ECU-MG-301 to Evaluate the Safety and Efficacy of Eculizumab in Subjects With Refractory Generalized Myasthenia Gravis (gMG)
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Secondary ID [1]
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2013-002191-41
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Secondary ID [2]
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ECU-MG-302
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Universal Trial Number (UTN)
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Trial acronym
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Linked study record
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Health condition
Health condition(s) or problem(s) studied:
Refractory Generalized Myasthenia Gravis
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Condition category
Condition code
Inflammatory and Immune System
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Autoimmune diseases
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Neurological
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Other neurological disorders
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Musculoskeletal
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Other muscular and skeletal disorders
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Intervention/exposure
Study type
Interventional
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Description of intervention(s) / exposure
Other interventions - Eculizumab
Treatment: Drugs - Placebo
Experimental: Eculizumab/Eculizumab - Blind Induction Phase: Participants who had received blinded treatment with eculizumab in Study ECU-MG-301 were administered eculizumab (4 vials/1200 milligrams [mg]) on Day 1 and Week 2 and placebo (4 vials/0 mg) at Weeks 1 and 3.
Open-Label Maintenance Phase: Participants received open-label eculizumab (4 vials/1200 mg) every 2 weeks starting at Week 4 and continued throughout the study.
Eculizumab 1200 mg was administered for up to 4 years in this extension study.
Experimental: Placebo/Eculizumab - Blind Induction Phase: Participants who had received blinded treatment with placebo in Study ECU-MG-301 were administered eculizumab/placebo (3 vials/900 mg, plus 1 vial/0 mg, respectively) on Day 1 and Weeks 1 through 3.
Open-Label Maintenance Phase: Participants received open-label eculizumab (4 vials/1200 mg) every 2 weeks starting at Week 4 and continued throughout the study.
Eculizumab 1200 mg was administered for up to 4 years in this extension study.
Other interventions: Eculizumab
Intravenous administration of eculizumab.
Treatment: Drugs: Placebo
Intravenous administration of matching placebo. Participants received placebo only during the Blind Induction Phase.
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Intervention code [1]
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Other interventions
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Treatment: Drugs
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Comparator / control treatment
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Control group
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Outcomes
Primary outcome [1]
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Count Of Participants With Treatment-Emergent Adverse Events
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Assessment method [1]
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Treatment-emergent adverse events (TEAEs) are adverse events with onset on or after the first study drug dose in Study ECU-MG-302. Likewise, treatment-emergent serious adverse events (TESAEs) are serious adverse events that onset on or after the first study drug dose in Study ECU-MG-302. A summary of serious and all other non-serious adverse events, regardless of causality, is located in the Reported Adverse Events module.
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Timepoint [1]
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Day 1 (after dosing) through End of Study (Week 208)
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Secondary outcome [1]
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Change From Baseline In Myasthenia Gravis Activities Of Daily Living Profile (MG-ADL) Total Score At Week 4 And Week 130
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Assessment method [1]
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The MG-ADL scale is a validated 8-item patient-reported outcome measure. Participants assessed their functional disability secondary to ocular (2 items), bulbar (3 items), respiratory (1 item), and gross motor or limb impairment (2 items). These 8 items were not weighted and were individually graded from 0 (normal) to 3 (most severe), providing a total MG-ADL score ranging from 0 to 24 points. A reduction in score indicates improvement in condition. Baseline was defined as the last available assessment prior to treatment (first study drug infusion) with eculizumab in Study ECU-MG-302. Change from Baseline in MG-ADL total score at Week 4 (blind induction phase) and at Week 130 (open-label eculizumab phase) are presented.
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Timepoint [1]
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Baseline, Week 4 and Week 130
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Eligibility
Key inclusion criteria
1. Participant has completed Study ECU-MG-301.
2. Participant has given written informed consent.
3. Participant was willing and able to comply with the protocol requirements for the
duration of the study.
4. Female participant of childbearing potential must have had a negative pregnancy test
(serum human chorionic gonadotropin). All participants were required to practice an
effective, reliable, and medically approved contraceptive regimen during the study and
for up to 5 months following discontinuation of treatment.
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Minimum age
18
Years
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Maximum age
No limit
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Sex
Both males and females
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Can healthy volunteers participate?
No
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Key exclusion criteria
1. Participants who withdrew from Study ECU-MG-301 as a result of an adverse event
related to study drug.
2. Female participants who were pregnant, breastfeeding, or intended to conceive during
the course of the study.
3. Unresolved meningococcal infection
4. Hypersensitivity to murine proteins or to one of the excipients of eculizumab
5. Any medical condition or circumstances that, in the opinion of the investigator, might
have interfered with the participant's participation in the study, posed any added
risk for the participant, or confounded the assessment of the participants.
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Study design
Purpose of the study
Treatment
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Allocation to intervention
Non-randomised trial
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Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
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Methods used to generate the sequence in which subjects will be randomised (sequence generation)
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Masking / blinding
Open (masking not used)
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Who is / are masked / blinded?
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Intervention assignment
Parallel
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Other design features
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Phase
Phase 3
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Type of endpoint/s
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Statistical methods / analysis
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Recruitment
Recruitment status
Completed
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Data analysis
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Reason for early stopping/withdrawal
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Other reasons
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Date of first participant enrolment
Anticipated
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Actual
12/11/2014
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Date of last participant enrolment
Anticipated
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Actual
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Date of last data collection
Anticipated
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Actual
15/01/2019
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Sample size
Target
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Accrual to date
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Final
117
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Recruitment in Australia
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Funding & Sponsors
Primary sponsor type
Commercial sector/Industry
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Name
Alexion Pharmaceuticals, Inc.
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Ethics approval
Ethics application status
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Summary
Brief summary
To evaluate the safety and efficacy of eculizumab in the treatment of refractory generalized
myasthenia gravis (gMG) as an extension study for the participants who previously completed
Study ECU-MG-301(NCT01997229).
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Trial website
https://clinicaltrials.gov/ct2/show/NCT02301624
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Trial related presentations / publications
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Public notes
This record is viewable in the ANZCTR as it had previously listed Australia and/or New Zealand as a recruitment site, however these sites have since been removed
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Contacts
Principal investigator
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Marcus Yountz, MD
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Address
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Alexion Pharmaceuticals, Inc.
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Summary Results
For IPD and results data, please see
https://clinicaltrials.gov/ct2/show/NCT02301624
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